Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome

Abstract

Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤.003) and psychosocial (P <.001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P <.001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P <.0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P <.04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis. Conclusions: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.

title = "Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome",

abstract = "Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤.003) and psychosocial (P <.001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P <.001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P <.0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P <.04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis. Conclusions: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.",

T1 - Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome

AU - Pediatric Heart Network Investigators

AU - New England Research Institutes

AU - Handisides, Jill C.

AU - Hollenbeck-Pringle, Danielle

AU - Uzark, Karen

AU - Trachtenberg, Felicia L.

AU - Pemberton, Victoria L.

AU - Atz, Teresa W.

AU - Bradley, Timothy J.

AU - Cappella, Elizabeth

AU - De Nobele, Sylvia

AU - Groh, Georgeann Keh Teng

AU - Hamstra, Michelle S.

AU - Korsin, Rosalind

AU - Levine, Jami C.

AU - Lindauer, Bergen

AU - Liou, Aimee

AU - Neal, Meghan K.Mac

AU - Markham, Larry W.

AU - Morrison, Tonia

AU - Mussatto, Kathleen A.

AU - Olson, Aaron K.

AU - Pierpont, Mary Ella M.

AU - Pyeritz, Reed E.

AU - Radojewski, Elizabeth A.

AU - Roman, Mary J.

AU - Xu, Mingfen

AU - Lacro, Ronald V.

AU - Pearson, Gail

AU - Stylianou, Mario

AU - Mahony, Lynn

AU - Sleeper, Lynn

AU - Tennstedt, Sharon

AU - Colan, Steven

AU - Klein, Gloria

AU - Guey, Lin

AU - Wruck, Lisa

AU - Travison, Thomas

AU - Chen, Shan

AU - Gerstenberger, Eric

AU - Olesker, Tanya

AU - Teitel, David F.

AU - Newburger, Jane

AU - King, Martha

AU - Dunbar-Masterson, Carolyn

AU - Posa, Andrea

AU - Nang, Quincy

AU - Hass, Cara

AU - Hsu, Daphne

AU - Lai, Wyman

AU - Hellenbrand, William

AU - Printz, Beth

PY - 2019/1

Y1 - 2019/1

N2 - Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤.003) and psychosocial (P <.001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P <.001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P <.0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P <.04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis. Conclusions: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.

AB - Objective: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. Study design: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. Results: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤.003) and psychosocial (P <.001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P <.001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P <.0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P <.04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis. Conclusions: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.